P4A Let's Talk Rare: The Life Science Podcast

In this episode of P4A Let's Talk Rare, hosts Georgie and Owen Bryant from Partners For Access are joined by Wes Michael, founder and president ofRare Patient Voice LLC. Wes shares the organization’s journey from its beginnings as a community project for hemophilia patients to a global platform that connects rare disease patients and caregivers with healthcare professionals. Rare Patient Voice has awarded over $30 million to participants and completed more than 210,000 projects, with Wes highlighting the company’s organic growth through patient referrals and advocacy partnerships.

Wes discusses the challenges of reaching patients nationwide, the rewarding impact of patient involvement in healthcare, and the essential role of patient-centricity in shaping drug development and health technology assessments. He emphasizes that patients' voices, as the end-users, are critical for creating effective healthcare solutions. Looking ahead, Wes shares his enthusiasm for expanding Rare Patient Voice into clinical trials and increasing its presence in Europe.

The episode concludes with a call to action for patients and caregivers to join the Rare Patient Voice community and share personal stories to further understanding and awareness of rare diseases.

Wes Michael Bio:

A market research industry veteran of over 40 years, Wes earned his MBA from the University of Chicago Booth School of Business and a BA in English from the University of Pennsylvania. In 2013, Wes launched Rare Patient Voice, based on a novel approach to building a market research panel that enables patients and caregivers to share their voices with researchers and companies developing products to improve patients’ lives. He structured the company on the core values of being a good patient resource, a good client partner, a good employer to staff, and a good citizen in our community. Since then, Wes has seen the company grow, expanding from the US to Canada, the United Kingdom, Spain, Italy, France, and Germany, completing thousands of projects, and providing customized service to hundreds of clients. He hopes that RPV’s work can help the lives of patients and caregivers who have participated in our studies and those who come after.

Episode Resources:

Wes Michael on LinkedIn

Rare Patient Voice LLC Website

Owen Bryant on LinkedIn

Georgina Rack on LinkedIn

Partners4Access Website 

P4A Let’s Talk Rare podcast on Apple Podcasts

Welcome to this month's episode of the P4A Let’s Talk Rare podcast by Partners4Acess. Today, Georgie and Owen are joined by their colleagues Aurelija Luko, the CFO, and Darren Callanan, the Global Digital Lead at Partners4Access. Join them as they discuss the recent job cuts in the pharma and biotech industry and the reasons behind them.

Aurelia explains that redundancies are not limited to the pharma industry and are influenced by factors like mergers, expiring patents, and macroeconomic instability. Darren adds that high drug development costs and pricing pressures also contribute to layoffs. However, he suggests that companies can use digital tools to enhance efficiency and innovation to address industry challenges. They also discuss a digital launch platform and a social listening tool created by Partners4Access. 

Aurelia highlights that companies are looking to increase shareholder value and reduce costs. Darren adds that thanks to the high cost of drug development and downward pressure on prices, companies are forced to narrow their focus and compete in the same space, leading to layoffs.

The conversation then shifts to discuss the digital launch platform created by Partners for Access. Darren explains that the platform streamlines the access and utilization of launch materials, ensuring that affiliates have access to the most up-to-date and compliant versions. This helps overcome the challenge of inconsistent distribution and saves time for the teams. 

Darren also mentions another tool called RarePulse, a social listening platform. This tool collects and analyzes data from social media and other sources to provide insights on key opinion leaders, unmet patient needs, and other relevant topics. He emphasizes that social media listening allows for a broader understanding of patient experiences and can help shape primary research and launch strategies.

Aurelija Luko Bio:

Aurelija’s work at P4A includes overseeing all accounting activities, from managing financial risk to setting budgets. Also, to ensure smooth day-to-day finance operations within the business.​ Aurelija is a Chartered Accountant with 10+ years of experience; she holds an MSc in Financial Economics from the University of Exeter and a BSc in the same field. ​

​Before P4A, she worked for small start-ups and big listed companies in healthcare, consulting, and property industries. She is a problem solver who can adapt well to new situations and review and improve the internal controls within the organization.

Darren Callanan Bio:

Darren Callanan is the Global Digital Lead at Partners4Access. He works in a specialist consultancy team to develop digital solutions addressing healthcare delivery barriers. Darren holds a master’s degree in Biotechnology and Business Management from the University of Warwick and an honors degree in Physiology from the National University of Ireland, Galway. His expertise includes strong communication and interpersonal skills, supported by a robust scientific background across various therapeutic areas. Previously, he worked at S3 Connected Health.

Episode Resources:

Aurelija Luko on LinkedIn

Darren Callanan on LinkedIn

Owen Bryant on LinkedIn

Georgina Rack on LinkedIn

Partners4Access Website 

P4A Let’s Talk Rare podcast on Apple Podcasts

Welcome to this month's episode of the P4A Let’s Talk Rare podcast by Partners4Acess. Today, Georgie and Owen are joined by their colleagues Pina Haberl, Senior Director, and Sam Morrison, Executive Director at Partners4Access. Join us as we discuss the crucial role of patient experience data in drug development. 

The conversation explores the need for drug developers to understand patient perspectives and gather data on their experiences, preferences, and needs. Pina and Sam highlight how patient experience data can improve drug development and clinical trial design while differentiating products in the market. They also address challenges in collecting and integrating patient data and the importance of involving patients early in the process. 

Also discussed is how some regulators and health technology assessment (HTA) bodies have started to accept patient experience data in their decision-making processes, but there is still uncertainty and inconsistency in how it is used. 

There is a call for more collaboration among stakeholders, including payers, policymakers, and patient groups, to establish best practices for collecting, analyzing, and utilizing patient experience data. Pina emphasizes the need for patients to have a seat at the table and be heard in decision-making processes. She mentions initiatives, such as partnering with patients and conferences, but acknowledges that there is still a long way to go.

A study published in Cambridge University Press investigated the utilization of patient preference (PP) data in health technology assessment (HTA) bodies worldwide. An exploratory survey by the HTA International Patient and Citizen Involvement Interest Group gathered insights from 40 members across twelve countries. Results showed that 82.5% of respondents formally considered PP data in HTA reviews, yet only 39.4% reported its frequent inclusion in assessments due to time and resource limitations. 

PP data moderately influenced decision-making processes and outcomes, with a higher impact on decision quality. Participants suggested patient advocacy groups should play a primary role in generating and submitting such evidence. The study highlights barriers to broader integration and proposes collaborative workshops for further investigation.

Pina (Josephina) Haberl Bio:

Pina is a goal-driven, solution-oriented, hands-on, and team-spirited professional with over 10 years of proven Market Access in consulting and industry expertise. She thrives on teamwork and excels at delivering actionable and innovative solutions to succeed in meeting business challenges. 

She specializes in developing and executing Market Access strategies and tactics that ensure success for pharmaceuticals, medical devices, and diagnostics in an international, cross-functional environment. Her understanding of the intricacies and specificities of respective healthcare systems and the diplomatic needs of diverse teams is critical in delivering results and ensuring patients have access to innovations.

Sam Morrison Bio:

Sam is an Executive Director at Partners4Access (P4A) with over 15 years of experience in market access consultancy. She has gained considerable insight into health systems across Europe and the USA, as well as other major markets, including Japan, China, and Brazil, as well as many Central and Eastern European markets. Sam has conducted many market access due diligence projects, and as pricing and reimbursement have become more essential to the licensing process, she is used to the time pressures and the balance between commercial, clinical development, and market access these projects require.

Episode Resources:

Welcome to this month's episode of Let’s Talk Rare: The Life Science Podcast brought to you by Partners4Access. Host Georgie records this episode at the World EPA Conference in Amsterdam. She holds a panel discussion with Juliette Sinclair-Spence, Sandrine Ruiz, 

Neil Grubert, and Seema Sondhi, and together we shared our experiences at the EPA conference. 

We also shared our excitement for AI and patient experience topics, as well as the importance of sustainability and finding new pricing approaches in healthcare. Gain insights on inclusivity and involving patients in drug development. Get ready for a meaningful, engaging conversation that will leave you inspired.

Take a deep dive into the benefits of tiered pricing, the challenges and concerns of companies and HTA agencies in joint HTA assessments, the importance of inclusivity, and the importance of involving and educating others.

Juliette Sinclair-Spence: Bio and Quote

LinkedIn

Juliette is the Funder and Chairman of the AK Eye Foundation and is a committed 𝐀𝐜𝐚𝐧𝐭𝐡𝐚𝐦𝐨𝐞𝐛𝐚 𝐊𝐞𝐫𝐚𝐭𝐢𝐭𝐢𝐬 (𝐀𝐊) 𝐏𝐚𝐭𝐢𝐞𝐧𝐭 𝐀𝐝𝐯𝐨𝐜𝐚𝐭𝐞. She has experienced this rare eye disease, and her key learning was that there is much that needs to be done on many fronts, including prevention, education, diagnosis, treatment, research, and support. Her passion for fighting against the disease led her to set up the first worldwide foundation focused on Acanthamoeba Keratitis.

 “I think involving patients in the drug development process is so important. We need to hear their voices and understand their journeys.”

Sandrine Ruiz: Bio and Quote

LinkedIn

Sandrine, or Sandy as she likes to be called, is the Senior Director of MarketAccess, Pricing, and HEOR at Immunocore. She is an experienced Pricing & Market Access professional with 25+ years in the biopharmaceutical industry. She has hands-on experience in giving strategic and science-based input across the pharmaceutical development workflow, from early stage to late stage, into Market/Patient Access, Pricing & Reimbursement, Payer Strategy, Real World Evidence, Clinical Development, Clinical Operations, and Business Development.

“I was really intrigued by the panel discussion on tiered pricing. It's such a hot topic right now, and it was interesting to hear the different perspectives on patient access. I'm also fascinated by digital information and the new trends in our field.”

Neil Grubert: Bio and Quote

LinkedIn

Neil is an Independent Global Market Access Consultant and a multilingual pharmaceutical market access specialist with 30 years of experience tracking the global prescription drug and self-medication markets. He has spearheaded the establishment and growth of Decision Resources’ international market access business. As the author of more than 150 reports covering 20 mature and emerging markets, multiple therapeutic areas, and numerous industry issues, he has earned a reputation for extensive knowledge of market access environments around the world.

“Yes, tiered pricing is definitely a challenging issue. Speaking of challenges, the EU HTA implementation has been causing many uncertainties for companies and HTA agencies. We need to navigate it carefully.”

Seema Sondhi: Bio and Quote

LinkedIn

Seema is the Head of Payer Value and Access for the Vaccine Business Unit at Takeda. She is passionate about patient access to innovative medicines and has worked diligently in her country, regional, and global roles to ensure access to pharmaceuticals, vaccines, medical devices, and orphan drugs. This has been achieved through key activities such as influencing global clinical trial development to ensure the collection of appropriate evidence, all the way through to launching products at prices that allow good levels of patient access and ensuring continued access post-launch via mechanisms such as innovative access schemes.

“Inclusivity is such an important aspect of our work. We can't just think about it; we have to be involved and educate others actively. Patients need to understand the questions asked and the impact of their input.”

Welcome to this month's episode of the Let’s Talk Rare: The Life Science Podcast by Partners4Acess. Georgie and Owen are joined by Nick Meade, Head of Policy at Genetic Alliance, to discuss the challenges faced by rare patients in accessing life-saving medicines.

Together they explore the EU joint HTA legislation coming into force in January 2025, and the importance of patient experience data. Nick explains that the challenges for rare patients remain the same, with diagnosis being the first hurdle. He highlights the progress being made in genetic diagnoses and screening but emphasizes the need for more harmonization in Europe. 

Finally, they touch on the concept of patient experience data, which refers to the inclusion of patient perspectives and outcomes in decision-making processes. Nick emphasizes the importance of this data in understanding rare conditions and leveraging it for better access to treatments. They also discuss the various events and initiatives planned by Genetic Alliance for  the upcoming Rare Disease Day on February 29th, to raise awareness and share stories of rare conditions.

Nick Meade Bio:

Nick Meade is Head of Policy at Genetic Alliance, the largest alliance of organizations supporting people with genetic, rare and undiagnosed conditions in the UK. He has over twenty years of experience in the field and is a member of the NIHR Advanced Therapy Medicinal Product (ATMP) Group. 

Episode Resources:

Nick Meade on LinkedIn

Genetic Alliance website

Partners4Access website  

P4A Let’s Talk Rare podcast on Apple

Hello and welcome to this special episode of Let’s Talk Rare: The Life Science Podcast as we celebrate 5 years of bringing you the podcast that is now the number one life science podcast across all platforms. We at Partners 4 Access want to thank every single one of our 35,000 subscribers and all the guests who have graced the podcast from the bottom of our hearts, we would not have gotten here without you. 

A special episode calls for special guests, and joining us today are our in-house expert and Managing Partner at P4A, Sophie Schmitz, our host from 2018-2021, Aparna Krishnan, and Prasan Subedi, Access Strategy Team Lead at Pfizer. Together, we walk down memory lane and relive the high points and challenges of the past five years, and share insights on rare diseases and cell and gene therapies.

Aparna Krishnan Bio:

Aparna has over 10 years of experience in the pharmaceutical and healthcare industry. As part of the senior leadership team, her primary focus is on strategic corporate engagement, legal, financial operations, and human resources. She has previously worked as Manager of Life Sciences at IHS Markit and as Consultant at Parexel’s Commercialization unit, among others specializing in market access, pricing, and reimbursement.

Sophie Schmitz Bio:

Sophie is the managing partner at Partners 4 Access, a company that believes in a sustainable rare disease world, which means helping our clients unlock the true opportunity of orphan drugs and ATMPs. Her 20+ year career spans various disease areas and medical devices, successfully supporting the strategy development and operationalization for orphan drugs. She brings a wealth of commercial and access experience across orphan and cell and gene therapies to the benefit of clients. She plays an active role in advising clients on price and market access strategies, with the ultimate goal of optimizing fair access for all stakeholders in rare diseases.

In this podcast, Georgie, RJ, and Owen discuss revolutionizing patient outcomes and the power of digital health solutions. RJ, a healthcare industry expert, shares insights on the role of technology in improving patient care and the future of digital health. They also touch on RJ's involvement in triathlons and his passion for running. With a friendly and engaging tone, this podcast offers valuable insights into healthcare and the potential of digital solutions.

RJ Kedziora Bio:

Mr. Kedziora is the co-founder of Estenda Solutions, a leading company specializing in custom software and data analysis for healthcare and medical companies. With a remarkable journey spanning over 30 years, he possesses a deep understanding of designing, developing, and deploying successful software projects. His extensive experience enables him to provide valuable guidance and innovative insights, resulting in cost-effective solutions that improve patient outcomes.

Mr. Kedziora received his M.B.A. from West Chester University and a Bachelor of Science in Computer Science from Duquesne University where he received the Excellence in Computer Science Award. He has spoken at numerous technical and healthcare conferences on a variety of topics and written or co-authored multiple articles focused on healthcare information technology, several published in peer-reviewed scientific journals.

Social Media Links:

FACEBOOK (BUSINESS): https://www.facebook.com/EstendaSolutions

LINKEDIN: https://www.linkedin.com/in/rjkedziora/

INSTAGRAM: https://www.instagram.com/estendasolutions/

TWITTER: https://twitter.com/Estenda

YOUTUBE: https://www.youtube.com/channel/UCuAfCbIFW0DmeI5PDS1PXcg

Welcome to this month's episode of the Let’s Talk Rare podcast by Partners4Acess. Georgie and Owen are joined by Iola Forster, Head of Publications and Portfolio at Karger Publishers, to discuss the future of patient engagement and the role of technology in healthcare. 

Together, they cover the future of HCPs (healthcare practitioners) and patient engagement, highlighting the importance of technology and patient empowerment in driving this evolution. They also emphasize the need for equitable access to healthcare information.

The conversation touches on the changing landscape of patient communication over the past fifteen years, with the advent of digital platforms and social media. They discuss how digitization has democratized access to health information and created opportunities for patients to connect with others facing similar conditions.

They further explore the impact of digitization on the orphan drug and cell and gene therapy world, emphasizing the role of patient involvement in content creation and the use of multimedia resources like infographics to cater to diverse learning styles.

Iola Forster Bio:

Iola Forster is the Head of Publications and Portfolio at Karger Publishers. She has over fifteen years of experience in the publishing industry and is skilled at fostering partnerships between the life sciences, healthcare clients, and Karger. Iola brings a wealth of knowledge and expertise to the table.

Episode Resources:

Iola Forster on LinkedIn

Karger Publishers website

Partners4Access website  

P4A Let’s Talk Rare podcast on Apple

Welcome to this month's episode of the Let’s Talk Rare podcast brought to you by Partners4Acess. Georgie and Owen are joined by Chloe Sheppard and Akshay Kumar to discuss the new EU HTA regulation set to be implemented in 2025. They explore the implications for drug developers of ATMPs, the attractiveness of the EU market, and the importance of proactive preparation for the upcoming changes. 

The topics covered include: key milestones of the EU HTA regulation; the importance of drug developers of ATMPs and oncology medicines to not adopt a wait and watch strategy; the risks of ignoring this change and the potential impact on clinical development plans, commercialization strategies, and patient access; the opportunities the EU market presents; and the need to start planning and adapting internal processes to successfully navigate the new regulatory landscape.

Chloe Sheppard Bio:

Chloe serves as a Senior Consultant at Partners4Access, working closely with clients to craft custom solutions for their pricing and market access obstacles. Her motivation lies in transforming the immense potential of advanced medicines into tangible therapeutic benefits for patients.

Akshay Kumar Bio:

Akshay, a Partner at Partners4Access, brings over 13 years of experience assisting pharmaceutical companies in devising market access and pricing strategies that align with their broader commercial and organizational contexts. His expertise spans not only product-level solutions but also process improvements and capability enhancement. In addition to market access, Akshay is well-versed in various other aspects of drug commercialization, including opportunity assessments, product positioning, forecasting, customer insights, and organizational design.

Episode Resources:

Chloe’s LinkedIn

Akshay’s LinkedIn

Partners 4 Access Website

In this episode we will be talking to Alejandro (Alex) Dorenbaum, M.D., CMO of Reneo Pharmaceuticals. Alex discusses how the company is developing drugs for patients with #rare mitochondrial diseases, a high unmet disease with no current treatments available. Their lead candidate #Mavodelpar has recently completed enrolment for their pivotal STRIDE clinical trial. STRIDE is a global, randomized double-blind, 6 months, placebo-controlled trial designed to assess the efficacy and safety of Mavodelpar.

Alex also discusses the importance of engaging early with #patients and patient organisations for #PMM across the world to truly understand the patient journey, their challenges & daily routines which help to shape the study design. This ensures successful enrolment and patient retention throughout the clinical trial, something Reneo have successfully completed throughout the development of Mavodelpar. By engaging early with patients, clinicians & regulators, they have completed enrolment in record time, this is especially difficult for #rarediseases as it is often difficult to find the patients and something Alex is extremely proud of.

Reneo Pharma: https://reneopharma.com/

Reneo is a clinical-stage pharmaceutical company focused on the development of therapies for patients with rare genetic diseases including mitochondrial diseases with significant unmet medical needs.

LinkedIn: Alejandro Dorenbaum

https://www.linkedin.com/in/alejandro-alex-dorenbaum-m-d-b01a6ab/

JustGiving: https://www.justgiving.com/fundraising/partners4access-b-v?utm_source=copyLink&utm_med[…]ampaign=pfp-share&utm_term=0c6d8180e0f3440b8383845adedb6513

P4A are joined by Xortx Therapeutics' CEO, Dr Allen Davidoff, as he discusses the journey from early development to potential launch for their novel therapy XRX-008 to treat patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD).

Allen discusses the company vision for developing novel therapies for rare progressive kidney disorders, and tells us about the launch journey for their main therapeutic candidate for AKPKD. How they managed to complete enrolment for late phase study in record time by engaging with patients and advocacy groups early, to really understand the patient journey and how the disease affects their quality of life. This helped to shape the clinical trial design by truly putting the patients at the forefront of all decisions and to ensure they were involved at all stages of the process.

Bio: Dr. Allen W. Davidoff, Ph.D. (15 years drug development experience ), Currently President and CEO of XORTX Therapeutics Inc (2013- ), Formerly, Chief Scientific Officer, VP Product Development and co-founder of Stem Cell Therapeutics Corp (Trillium Therapeutics- TRIL: NASDAQ) (7 yrs). Prior to Stem Cell Therapeutics Corp., Senior Scientist and Head of Pharmacology at Cardiome Pharma Corp. Dr. Davidoff has 12 + years of therapeutic drug development experience with a focus on clinical and regulatory developmental affairs, in the US, Canada, Germany, Denmark, and India. Senior management and leadership experience includes pharmaceutical non-clinical and clinical R&D including 2 investigational new drug (“IND”) applications or supplemental IND’s, 2 phase I studies (4 multi-country), 7 phase II studies, and 1 NDA.

Dr Allen Davidoff: https://www.linkedin.com/in/allen-davidoff-9a80968/

XORTX Therapeutics

XORTX Therapeutics, Inc. is a drug based biotechnology company primarily focused on orphan disease indications which have aberrant purine metabolism and frequently high serum uric acid imbalance. Our focus on developing kidney therapies also includes therapeutics programs for large markets such as diabetic nephropathy and acute kidney injury associated with respiratory virus infections such as SARS-CoV-2.

We possess patents and patent applications that may include U.S. and international rights regarding the development of uric acid-lowering agents for the treatment of hypertension, insulin resistance, diabetes, metabolic syndrome, and kidney injury.

XORTX Therapeutics: https://www.xortx.com/

P4A host a special episode live from World Evidence, Pricing & Access Conference 2023 in Amsterdam.

Join Georgie & Owen hosting live from World EPA Conference 2023. We will be bringing you speaker interviews, giving a flavour of the atmosphere and discussing some of the trends & key takeaways that came out of the conference.

Max Rex:

LinkedIn: https://www.linkedin.com/in/max-rex-7a364789/

Stefaan Friers:

Takeda: https://www.takeda.com/

LinkedIn: https://www.linkedin.com/in/stefaanfiers/

Juliette Sinclair Spence:

LinkedIn: https://www.linkedin.com/in/juliettevss/

Website: https://akeyefoundation.com/

Support Group: https://www.facebook.com/groups/acanthamoebakeratitissupportgroup

Donate: https://akeyefoundation.com/donation/

Youtube video sharing my story: https://youtu.be/l35c4CF9DwQ

Jelle Kleijn:

Website: https://www.sifigroup.com/

LinkedIn: https://www.linkedin.com/in/jelle-kleijn-b6bbb520/

In this episode of Let’s Talk Rare, as we commemorate World Rare Disease Day, Georgie and Owen are joined by Louise Fish and Dan Lewi to discuss all aspects surrounding rare diseases, from key challenges patients face in getting access to life-saving medicines, the clinical trial burden, EU pharmaceutical strategy and more.

The topics covered include: Why genetic screening of newborns in the UK lags behind the EU, funding rare disease research, clinical trial burden, patient registries, patient experience data and its relevance, revamping data sharing with families, and how pharma can involve patients earlier.

Louise Fish Bio:

Louise is a senior leader with over 25 years of executive and non-executive board experience in health and social care in charitable, public and commercial sectors. She is passionate about improving the NHS and social care services by listening to and learning from the experiences of patients and their families. She has a strong understanding of how to drive change.

Dan Lewi Bio:

Dan is the Chief Executive and co-founder of the only Tay-Sachs and Sandhoff disease-specific charity in the UK called The Cure & Action for Tay-Sachs (CATS) Foundation. Selected and appointed as the Chairman of the European Tay-Sachs and Sandhoff Charity Consortium (ETSCC), which has brought together all the Tay-Sachs and Sandhoff charities in Europe.

The P4A team have hand-picked trends to look out for in 2023. This episode we are joined by special guest Neil Grubert who will be discussing the topics in further detail, alongside our P4A experts, Shrishti & Jodie.

The topics covered are: EU Pharmaceutical Strategy, OMP Legislation, Joint HTA Assessments - cross-country HTA cooperation, Expansion of CAR-T & Hospital Exemptions across Europe and finally the Patient - Putting patients at the forefront of drug developers.

Neil Grubert Bio: 

Neil has worked in Industry for over 20 years starting at Decision Resources as a researcher in 1997 and leaving as Vice President of Global Market Access Insights in 2013. Currently he is an independent MA consultant and has been doing this for the last 8 years.

LinkedIn: https://www.linkedin.com/in/neil-grubert/

Hosts: Georgie Rack & Owen Bryant

P4A Speakers: Srishti Gupta, Senior Consultant & Jodie Lyons, Analyst

Produced by: Operations

Did you miss the World Orphan Drug Congress Europe 2022, held in Sitges, Barcelona in November? If you did, don't worry P4A have you covered!

This month's special  podcast was part recorded live at the conference and part recorded back in London with members of the team that attended! We will be speaking to a few presenters & sponsors to discuss their presentations, key learnings and their personal highlights from the conference. Also the P4A team will be talking about our favourite sessions and the key takeaways from the conference.

Special thanks to our guests for their insights and learnings throughout the podcast.

Presenters: Georgie Rack & Owen Bryant

Guests:

Alexander Natz, Secretary General at EUCOPE - https://www.eucope.org/

Leon van Wouwe, Clinical Innovation Director at VOLV Global https://www.volv.global/

Wing-yun Cheung, General Manager at Terrapinn & organiser of WODC EU - https://www.terrapinn.com/conference/world-orphan-drug-congress/index.stm

Chloe Sheppard, Consultant at P4A

Joanna Fernandes, Senior Consultant at P4A

 Winds of change are howling in Germany, with the draft healthcare bill now approved to stabilise SHI fund finances. What will manufacturers, with innovative orphan drugs and cell and gene therapies, launch strategies be?

With the latest decision from Janssen to avoid the German market altogether for x2 Rare Oncology innovative drugs, will this be a trend we are likely to see continue? 

Join Stefan Walzer & Fisentzos Stylianou discuss the new bill, in regards to the biggest changes that impact orphan drug (OD) manufacturers. Will OD manufacturers still see Germany as the first go to market within Europe and what does this means for rare disease patients?  Will there be delays to new treatments or will manufacturers decide not to launch in Germany at all to protect the price of their new drug? 

We will be discussing this and so much more! If you are a drug manufacturer planning your launch strategy, this podcast is for you! 

Presenters:

Georgie Rack & Owen Bryant

Guests

Stefan Walzer, CEO President & Founder of MArS Market Access & Pricing Strategy GmbH

Fisentzos Stylianou, senior analyst and P4A's German country expert

Dr Stefan Walzer, CEO President & Founder at MArS Market Access &Pricing Strategy GmbH

https://marketaccess-pricingstrategy.de/en/

Bio & Fun Facts 

1) Economist, PhD in health economics, diploma in clinical trials

2) Experience in MA, reimbursement, HE and pricing since 2004 in consultancy and industry

3) Founder and CEO of MArS - THE D-A-CH market access consultancy. Linked to that also co-founder of SMS2DACH including the full spectrum for D-A-CH support (distribution, management, launch, etc.): www.sms2dach.com

4) Founder of P&N (pricing-and-negotiations.ca) with a focus on negotiations across the world including being the co-founder of www.thenegotiationlab.com

5) Member of a European network Tesseract (https://www.tesseracteurope.com/) which can serve companies moving from outside Europe into Europe including not only reimbursement services but also logistics, customs, etc.

6) Teaching at various German universities

7) Love spending time with my family, being a soccer coach of under 14 years-old and being a supporter of Borussia Dortmund

Fisentzos Stylianou, Senior Analyst & German Country Expert

Fisentzos role at Partners4Access includes conducting primary and secondary research to support the development of market access and reimbursement strategies for clients in the pharmaceutical and biotechnology industries. With a passion for innovative treatments, he closely follows the cell and gene therapy field as it expands to treat more patients with rare diseases. 

Prior to joining Partners4Access, Fisentzos worked as a Research Associate at Imperial College London, where he also earned his Ph.D. in Structural Biology in 2020. During this time, he conducted research as part of a multidisciplinary team to advance his understanding of the structure and function of biofilm-forming proteins, paving the way for the design of novel antimicrobial therapeutics. Fisentzos also holds an M.Sc. in Biomedical and Molecular Sciences Research and a B.Sc. in Biomedical Science, both from King’s College London. 

P4A’s 2 part -Patient Empowerment podcast series has been released. Our incredible panel of experts Laurence Woollard, Neil Bertelsen & Sophie Schmitz are back with us to carry on the discussion in part 2 on true patient empowerment and really looking at the WHY’s! Why should drug manufacturers involve & listen to and involve the patients. What are the implications, if any, for drug manufacturers who do not include the patient voice. How can we bring all stakeholders together to work completely new approaches to medicine approval and patient access. Lastly, we will look at initiatives or services our panel are offering to try to bridge the gaps and barriers for sustainable patient access.

P4A’s’’Let’s talk rare’’ monthly podcasts are available wherever you listen to your podcasts. If you are a drug manufacturer and you haven’t yet listened, this needs to be at the top of your to do list!

If you missed out on part 1 - listen here: https://spotifyanchor-web.app.link/e/hOBI3UqkPtb

Hosts: Georgie Rack (G-Rack)  & Owen Bryant (OB1)

Produced by: Operations Team

Links:

P4A’s PCAC: https://partners4access.com/services/pc-council/

Part One: Patient Empowerment: https://spotifyanchor-web.app.link/e/hOBI3UqkPtb

ABPI article in recognition of Rare Disease Day 2022 - “Moving      beyond box-ticking and lip-service - why patient involvement matters in a      new era of ATMPs for rare diseases”

James Lind Alliance Priority Setting Partnership -

“Setting       priorities for bleeding disorders - Final report” (2018)

“More than a Top 10: How James Lind Alliance Priority Setting Partnerships transform research, people and organisations” (2019)

This month we have a special 2-part series focused on Patient Empowerment, with special guests Laurence Woollard, On the Pulse, Neil Bertelsen, Independent Consultant & Sophie Schmitz, Managing Partner at P4A. This podcast is a MUST to listen too if you are a company looking to successfully develop and commercialize an orphan drug or ATMP

The first part will focus on how do we ensure there is genuine patient involvement at all stages of drug development? What are the challenges and barriers for patients living with a rare disease vs drug developers within the rare/ultra-rare sphere?

The second part will solely focus on the WHY! Why should drug manufacturers, payors, regulators and HTA bodies involve and listen to the patients? What do they bring to the table? We will also dive into the implications of not including the patient voice and use examples of companies that have successfully managed to do this and the benefits of doing so.

Laurence Woollard, Owner On The Pulse

Laurence Woollard is founder and director of On The Pulse – an independent, strategic consultancy providing specialist insight to global healthcare providers and multi-agencies on the development and rollout of patient activation campaigns in haemophilia and rare disease. Laurence has extensive patient advocacy experience, having advised on and implemented a broad scope of community awareness, research and educational initiatives for commercial partners and third-sector organisations. 

Neil Bertelsen, Independent Consultant

Neil Bertelsen is an independent consultant and patient advocate with over 25 years of experience bringing the patient voice to health care decision makers and communicating the science of health care to patients in a way that truly informs their own personal health choices. Neil is passionate about bringing the patient experience and perspectives to decision-makers including industry and health technology assessment (HTA) bodies. Neil is the past chair of HTAi (the global scientific society of HTA) Patient and Citizen Involvement Interest Group. This is an international multi-stakeholder group whose remit is to work alongside HTA organizations and patient organizations to bring patient involvement processes and patient insights and experiences into access decision making processes.

Sophie Schmitz, Managing Partner P4A

Sophie is managing partner at Partners4Access(P4A),an award winning global consultancy focused on access for orphan drugs & ATMPS. P4A support the biotechnology & pharma industry along their launch journey to help secure successful price, reimbursement and access for orphan drugs & ATMPs. The company has solid partnerships with clients supporting their strategy & operations to effectively launch success. 

This month we have put together a slightly different episode with a twist. Georgie & Owen will take you behind the scenes at P4A and discuss 'What makes P4A an award-winning global market access consultancy?' Why we do what we do every day, and what it means to each and every member of staff. Pride, Passion & Partnership are the core values at P4A, and we take a closer look into each one. Later in the show we have a very special announcement from our Managing Partner, Sophie Schmitz.

Host: Georgie Rack, Communication Executive & Owen Bryant, Creative Director

Produced By: Operations

Awards P4A have won in 2022 (so far!) - https://partners4access.com/about/awards/

1. BOBI Awards 2022 - Analyst Team of the Year

Link: https://www.bhbia.org.uk/bobi-awards/award-winners/bobi-2022

2. Benelux Enterprise Awards 2022- Best Orphan Drug & Gene Therapy Experts

Link: https://www.eubusinessnews.com/awards/benelux-business-awards/#ourwinners

3. International Life Science Awards 2022 - Best Cell & Gene Therapy Global Market Access Consultancy

Link: https://www.ghp-news.com/awards/international-life-sciences-awards/

4. Healthcare & Pharmaceutical Awards 2022 - Leading Experts in Orphan Drug & Gene Therapy Access

Link: https://www.ghp-news.com/awards/healthcare-pharmaceuticals-awards/

In this episode Chloe & Alexander will be starting the discussion with an overview of EU HTA regulation, looking at the harmonization debate and how this came about. We then discuss JCA evaluation methodology (e.g. comparator selection, acceptability of clinical evidence), the potential new role of individual country agencies and how the joint HTA process will link to national processes. EUnetHTA21 and how the methodology is evolving with G-BA & IQWIG increasingly involved, will this be a major influence in the development of final methodology? Finally, we look at how Pharma companies should be best preparing for the implementation of the regulation.

Host: Chloe Sheppard

Guest Speaker: Alexander Natz

Produced by: Operations team

Helpful links:

#EUHTAPriorities – EUCOPE’s Five Priorities for a Successful EU HTA Procedure

Sounds of Science Podcast – Episode 1: A Future-Proof HTA system in Europe?

https://partners4access.com/whitepapers/pan-european-joint-hta-what-does-the-future-hold-for-innovative-therapies/

About Alex:

Alexander Natz is the Secretary General of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) in Brussels and advises innovative pharmaceutical and biotech companies, including start-ups, in regulatory and pricing & reimbursement matters from the EU law and German law perspective. From 2008 to 2013, he was Head of the Brussels Office of Bundesverband der Pharmazeutischen Industrie e.V. (BPI). Before, he worked as a lawyer at Sträter Law Firm in Germany with a special focus on managed entry agreements and licensing of pharmaceuticals. Dr. Natz has also worked in the field of competition law with the European Commission and in the pharmaceutical industry. As a research assistant at Duke University (USA) he has dealt with international pharmaceutical law. His doctorate was supervised by the former judge at the European Court of Justice, Prof. Dr. Dr. Ulrich Everling

Email: natz@eucope.org

LinkedIn: https://www.linkedin.com/in/alexander-natz-63467433/

About EUCOPE:

For more than a decade, the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) has been the voice of small to mid-sized health technology companies in Europe. As a trade association, we advocate for sound public policy that supports innovation, while fostering a community built on a shared purpose: improving and saving the lives of European patients through innovative therapies and medical technology.

Website: https://www.eucope.org/

Part 1 featuring Victor Maertens, Government Affairs Manager at EUCOPE

In this episode Victor and Aparna will be focusing on Advanced Therapy Medicinal Products (ATMPs). We will start by looking at the main challenges for manufacturers in the ATMP approval process and what is required from an HTA perspective. We then discuss EUCOPE’s position paper on the different payment models, including the motivation for the report and key learnings. We look at sustainable patient access and whether the report addresses the affordability issues surrounding ATMP’s drug pricing and if this is even possible in the current landscape. The EU’s revision of Blood, Tissue & Cells legislation and lastly the future of ATMPs for manufacturers and patients.

Host: Aparna Krishnan

Guest speaker: Victor Maertens

Produced by: Operations team

About Victor Maertens:

https://www.linkedin.com/in/victormaertens/

Victor Maertens is the Government Affairs Manager for the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE). Victor leads EUCOPE’s policy work on the Pharmaceutical Strategy and in the area of Cell and Gene Therapy. Prior to joining EUCOPE, Victor worked for Brussels-based consultancies, providing pharmaceutical companies, trade associations, and NGOs advocacy, strategy, and communications advice. His experience and interests include ATMPs, antimicrobial resistance, Orphan medicinal products (OMP), blood policy, and neglected tropical diseases.

About Eucope:

https://www.eucope.org/

For more than a decade, the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) has been the voice of small to mid-sized health technology companies in Europe. As a trade association, we advocate for sound public policy that supports innovation, while fostering a community built on a shared purpose: improving and saving the lives of European patients through innovative therapies and medical technology.

In 2020 China’s pharmaceutical market passed an important milestone, a Deloitte report cited that China was expected to grow its pharmaceutical market volume by 2020 to $220 billion (USD) becoming the second largest market behind the US. The commercial potential is huge, so what do we know about the orphan drug (OD) potential in China? Fisentzos Stylianou discusses China’s healthcare system, key challenges for OD manufacturers, Spinraza’s commercialisation journey in China, and incentives available to OD manufacturers in China.

Reference: https://www2.deloitte.com/cn/en/pages/life-sciences-and-healthcare/solutions/life-sciences.html

Presenter: Aparna Krishnan, Partner – Global Operations

Contributor: Fisentzos Stylianou, Analyst

Producer: Operations team

Over the last few years, the Middle East has piqued the interest of orphan drug manufacturers, and some have prioritised launching in this region before European markets. Akshay Kumar and Lavni Varyani discuss what is driving this behaviour, the orphan drug infrastructure within the region, and opportunities, barriers, and key considerations for manufacturers aiming to launch in the Middle East.

Presenter: Akshay Kumar, Partner, P4A

Contributor: Lavni Varyani, Founder, Pharma Business Partners

Producer: Operations team

For this Rare Disease Day episode, Janet and Nick discuss a range of topics associated with the rare disease community and patient advocacy. They address new-born screening, patient registries, the UK rare disease framework, the NICE methods review and much more!

Presenter: Georgie Rack, Communication Executive

Contributors: Janet Bloor, CEO of Duchenne Nexus Advocacy (DNA) and Nick Meade, Director of Policy and Joint Interim Chief Executive of Genetic Alliance UK

Producer: Operations team

Lots of anticipated changes are expected in the orphan drug, cell and gene therapy space and 2022 is going to be an exciting year. Listen to our latest podcast on the top trends in drug development, HTA and pricing from Akshay Kumar and Andrea Bernardini to help you navigate the year ahead.

Presenter: Georgie Rack

Speakers: Akshay Kumar & Andrea Bernardini

Produced by: Ops team

Sophie Schmitz and Joanna Fernandes discuss bluebird bio’s innovative gene therapy Zynteglo and its withdrawal from the European market. Sophie and Joanna will be looking at the fall of Zynteglo from two sides: from a company perspective and from the perspective of the EU environment.

Presenter: Georgie Rack, Communication Executive

Contributors: Sophie Schmitz, Managing Partner and Joanna Fernandes, Senior Consultant

Producer: Operations team

This episode featuring James Mackay, President & CEO of Aristea Therapeutics. James provides insights on his experience with setting up a biotech and challenges associated, industry climate on spin off opportunities, Aristea’s pipeline, collaborations and US PRMA reforms.

Presenter: Aparna Krishnan

Contributors: James Mackay, President & CEO of Aristea Therapeutics

Producer: Operations team

About Aristea Therapeutics, a San Diego-based clinical-stage immunology-focused drug development company developing novel therapies for serious, rare inflammatory diseases.

In this episode P4A’s Richard Wang and Adama Anozie discuss Hospital Exemption (HE) which is an emerging access pathway for Advanced Therapy Medicinal Products (ATMPs). Main topics of discussion include the benefits and potential downsides associated with this pathway, the EU regulations involving HE and recent events in Italy, Spain and France, as well as the implications of HE on the traditional pharma model, the need for collaboration between big pharma and hospitals/institutions to develop HE therapies and much more!

Presenter: Richard Wang

Contributor: Adama Anozie

Producer: Operations Team

In this episode P4A’s Senior Client Relationship Director, Bruce Chin discusses his experiences from the in person World Orphan Drug Congress USA 2021. Topics of discussion included travel and safety precautions due to COVID, Bruce’s favourite sessions, key learnings from the sessions, and much more!

Presenter: Georgie Rack

Contributor: Bruce Chin

Producer: Operations Team

In this episode Akshay Kumar and Richard Wang discuss how through innovative technology, decentralised clinical trials came to the limelight, facilitating remote access and ensuring continued operation of clinical trials. In addition, we discuss the future of decentralised clinical trials in a post-COVID world, its implications and demands within the industry.

Presenter: Akshay Kumar

Contributors: Richard Wang

Producer: Operations Team

In this episode, the P4A team take a deep dive into the Brazilian pharmaceutical market and discuss its potential for access to orphan drugs, cell and gene therapies. The overall Latin American pharma market is forecast to grow by 9% per year through to year 2028. However, major markets in this region such as Brazil have some major barriers to access. Listen in to learn more. 

Presenter and Contributor: Ciaran Cassidy

Producer: Aparna Krishnan

Its almost six months since President Joe Biden came to office but the role of the head of the country's key drug regulator, the US Food and Drug Administration (USFDA) is yet to be filled. In this episode, the P4A discusses the absence of a USFDA commissioner and its impact. Also, we understand the significance of recent remarks of the Center for Biologics Evaluation and Research (CBER) director Peter Marks on the need for consistency in the manufacture of cell and gene therapies. 

Presenter and Contributor: Max Rex

Producer: Aparna Krishnan

In this episode, P4A speaks to a special guest - Alliance of Regenerative Medicine's chief executive officer Janet Lambert on the ARM's key initiatives such as the Foundation of Cell and Gene Therapy medicines as well as the 2019 report on ATMPs (Advanced Therapy Medicinal Products).

Presenter: Aparna Krishnan

Contributor: Janet Lambert, CEO of Alliance of Regenerative Medicine

Producer: Aparna Krishnan   

In this month's episode, the P4A team take a deep dive into the impact of Brexit on the UK's market access landscape. Four months after leaving the European Union, the UK's attractiveness as a key destination for commercialisation of new drugs is being tested. In order to improve its credentials, the UK is exploring a slew of measures that could potentially spark a reform. 

But will that happen? P4A's Joanna Fernandes and Jayne Watson consider two distinct initiatives - MHRA's (Medicines Healthcare Regulatory Agency's) new I-LAP (Innovative Licensing and Access Pathway) scheme and England's HTA body NICE's (National Institute for Health and Care Excellence's) method review. 

Presenter and Contributor: Joanna Fernandes

Other contributors: Jayne Watson

Producer: Aparna Krishnan

The world of orphan drugs is at the cusp of a transformation in the post COVID era. In this episode, the team discuss the impact of the pandemic on value demonstration of orphan drugs. In order to be successful, manufacturers of precision medicine will need to focus on carving out a niche for their drugs and support health systems to find the right patient for their treatment at the right time. 

The team also explore the role of digital tools such as Artificial Intelligence (AI) and telemedicine in helping drugmakers optimise manufacturing and treatment delivery. This combined with a multi-stakeholder approach will provide the recipe for success for next generation`niche-busters'.

Presenter and Contributor : Akshay Kumar

Other contributors: Richard Wang, Andrea Bernardini and Erfan Akbraian

Producer: Aparna Krishnan

In this episode, the P4A team discussed the impact of cost-containment measures in Germany, the European Commission's recent move to revise legislation on orphan medicinal products and potential legislative reforms in the US under the new Biden administration. 

Presenter: Jens Leutloff

Contributors: Chloe Sheppard, Max Rex

Producer: Aparna Krishnan

In this Rare Disease Day special episode, we focus on the impact of COVID -19 on rare disease patients. Some of the key challenges for patients during this pandemic include disruption in their access to treatments, struggle to continue participation in clinical trials, lack of access to vital equipment such as PPE (Personal Protective Equipment) and also to healthcare personnel. We speak to Genetic Alliance chief executive officer Jayne Spink, patient Nicola Whitehill and Danielle Myers, mother and carer of patient 10 year old Dylan Myers.

Presenter: Aparna Krishnan

Contributors: Jayne Spink, Nicola Whitehill and Danielle Myers

Producer: Aparna Krishnan

More information on :

Genetic Alliance can be found at https://geneticalliance.org.uk/

Nicola Whitehill's blog: https://blog.raynaudsscleroderma.co.uk/2017/04/scleroderma-raynauds-rare-disease.html?m=1 

Dylan Myers' story: https://m.facebook.com/dylansstory/  and https://www.treeofhope.org.uk/dylansstory/

In this episode,  the P4A team review all the key events of the year 2020 in the orphan drug, cell and gene therapy world. The emergence of COVID-19 pandemic has caused disruption but also opportunities for the biotech industry. From  change in regulations, new stakeholder collaborations to impact of Brexit and US drug pricing reforms,  a full round up of 2020. So do listen in!

Presenter and Contributors: Sophie Schmitz  and Akshay Kumar

Producer: Aparna Krishnan

This episode discusses patient access to AveXis' gene therapy Zolgensma from Orsini Healthcare, US based specialty pharmacy's perspective.  The senior team at Orsini  provide insight on their experience of Zolgensma access including key manufacturer criteria, payer landscape along with challenges and lessons learnt. 

Presenter: Aparna Krishnan

Contributors: Dave Frobel, Senior Vice President, Trade & Tom Shaughnessy, Senior Vice President, Health Plans    

Producer: Aparna Krishnan

About Orsini Healthcare: Founded in 2009, Orsini is a leading independent specialty pharmacy focused on rare conditions and gene therapies. Orsini was one of the first specialty pharmacies to provide Zolgensma, the first gene therapy for Spinal Muscular Atrophy. Today, Orsini has access to 40 limited distribution drugs, has more than 525 direct payor contracts and is recognized as a reimbursement expert in managing both medical and pharmacy benefits.

This week is a special episode celebrating Rare Disease Day 2020.  Our guest speaker is Annie Kennedy, head of policy and advocacy at Every Life Foundation, a US based non profit organisation that works to advance the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy. Here she speaks about the organisation's initiatives on drug access. 

To know more about the foundation, visit https://everylifefoundation.org/ 

Presenter and Producer: Aparna Krishnan

Contributor: Annie Kennedy, Chief of Policy & Advocacy, EveryLife Foundation for Rare Diseases

As 2019 winds down, P4A's Sophie Schmitz and Akshay Kumar discuss the key trends that defined this year  -  marketing approval of cell and gene therapies; pricing and reimbursement challenges in US & EU5 as well as industry merger and acquisitions. 

Presenter: Sophie Schmitz

Contributor: Akshay Kumar

Producer: Aparna Krishnan

The team discuss the latest update on the proposed EU health technology assessment (HTA) regulation. The key objectives of the 2018 proposal was to promote convergence in HTA tools, procedures and methodologies; reduce duplication of efforts for HTA bodies and industry and improve joint use of outputs. This episode goes indepth into the one of the controversial aspects of the proposal - The joint clinical assessment - that caused a near stand still in negotiations between member states. 

Presenter: Christina Poschen

Contributor: Ciaran Cassidy

Producer: Aparna Krishnan

The team discuss the NORD summit particularly FDA commissioner Scott Gottleib’s presentation, panel discussion on cell and gene therapy pricing as well as P4A’s lunch and learn on cell and gene therapy access – learnings from the EU.

Also in this episode, Novartis’ Kymriah innovative payment agreement in Italy, the changing evaluation trends for gene therapies among HTA bodies and Alliance for Regenerative Medicines’ Q3 report.

Presenter: Jack Rawson

Contributor: Sophie Schmitz

Producer: Aparna Krishnan

Following an autumn hiatus, the team come back to discuss Vertex's journey to commercialize Orkambi picking up the story from the latest deal struck with NHS England in October 2019. 

Presenter: Nicola Allen

Contributor: Joanna Fernandes

Producer: Aparna Krishnan

The team takes a look at the most recent legislation in Germany known as GSAV or the law for more safety in the supply of pharmaceuticals and its implications on the access to orphan drugs. GSAV was approved by the German cabinet on January 30, 2019 and is due to come into effect in August this year. 

Presenter: Akshay Kumar

Contributor: Joanna Fernandes

Producer: Aparna Krishnan

This week, the P4A team discuss the coverage of Novartis' Zolgensma in the US in particular how US health insurers have received the gene therapy. Also on the agenda was the impact of Zolgensma's launch on rival Biogen's Spinraza and the potential for dominance of the gene therapy in the spinal muscular atrophy landscape. 

Presenter: Aparna Krishnan

Contributor: Max Rex

Producer: Aparna Krishnan

The debate over US drug pricing reform has reached fever pitch as the US Department of Health and Human Sciences announced its latest Medicare Part B proposal to include international reference pricing. The Partners4Access team looks at the background to this proposal; the political and patient organisation criticisms; the potential industry response and its ramifications. 

Presenter: Aparna Krishnan

Contributor: Max Rex

Producer: Aparna Krishnan

This week, we analyze Bluebirdbio's commercialization plans for its gene therapy Zynteglo including the new payment model and a delay in launch due to manufacturing issues. 

Presenter: Aparna Krishnan

Contributor: Joanna Fernandes

Producer: Aparna Krishnan

This week, the team discuss the emergence of 'drug buyer's clubs' in the UK involving Vertex's cystic fibrosis drug Orkambi. 

Presenter: Aparna Krishnan

Contributors: Sophie Schmitz, Ciaran Cassidy

Producer: Aparna Krishnan

This week, the team discuss Vertex's acquisition of Exonics and expansion of its collaboration with CRISPR Therapeutics. Also, we take an indepth look at the European Medicines Agency's approval of Bluebirdbio's gene therapy Zynteglo as a treatment for beta-thalassemia . 

Presenter: Aparna Krishnan

Contributor: Jack Rawson

Producer: Aparna Krishnan

On our anniversary episode, the P4A team discuss the USFDA approval of Novartis' Zolgensma, a one time gene therapy for paediatric spinal muscular atrophy patients and a look back at the last 12 months of our podcasting journey. 

Presenter: Joanna Fernandes

Contributor: Christina Poschen, Aparna Krishnan

Producer: Aparna Krishnan

In this episode, we look at Novartis CEO Vas Narasimhan calling for a change in the US drug payment systems, arguing for new economic models to identify how much value a cure represents. Also, Italy's attempts at introducing a draft resolution to improve transparency in drug pricing at the World Health Assembly.

Presenter: Aparna Krishnan

Contributors: Joanna Fernandes, Christina Poschen

Producer: Aparna Krishnan

This week, the team discuss the deal between UK's National Health Service and Biogen prompting the successful reimbursement of Biogen’s Spinraza as a treatment for spinal muscular atrophy and Solid Biosciences’ clinical update on its Duchenne Muscular Dystrophy gene therapy product SGT-001.  

Presenter: Aparna Krishnan

Contributor: Max Rex

Producer: Aparna Krishnan

This week, the P4A team discuss a new experimental therapy - Phage therapy or genetically engineered phages in order to treat bacterial infections and the acquisition of Theracon by Big Pharma firm Pfizer for upto $810 million.

Presenter: Aparna Krishnan

Contributor: Ciaran Cassidy

Producer: Aparna Krishnan

This week, we are looking at The Institute for Clinical and Economic Review or ICER’s white paper on alternative options for the US rebate system, and the establishment of a national agency to evaluate drug effectiveness and negotiate prices in Canada. 

Presenter: Max Rex

Contributor and Producer: Aparna Krishnan

This week, we feature a special guest podcast speaker - Scott  Dorfman, chief executive officer of non-profit gene therapy developer Odylia Therapeutics on his personal journey and the future in gene therapy development. 

If you would like to know more about Odylia Therapeutics, please visit  https://odylia.org/ 

Presenter: Aparna Krishnan

Contributor: Scott Dorfman, CEO, Odylia Therapeutics

Producer: Aparna Krishnan

This week, we look at the European Federation of Pharmaceutical Industries and Associations or EFPIA’s patient wait survey and its key findings as well as news developments in the spinal muscular atrophy world relating to gene therapy Novartis’ Zolgensma and Biogen’s Spinraza.

Presenter: Aparna Krishnan

Contributors: Sophie Schmitz, Jack Rawson

Producer: Aparna Krishnan

 As part of the continuing World Orphan Drug Congress (WODC) special, this episode discusses the cGMP facilities and biosafety solutions from Germfree’s Carol Houts, P4A’s Sophie Schmitz looks back at the 2019 conference and Terrapinn’s Andre Singer talks about what to expect from WODC 2020.

 Presenter and Producer: Aparna Krishnan

Contributors: Carol Houts, Director of Regulatory and Quality, Germfree; Sophie Schmitz, Managing Partner, Partners4Access and Andre Singer,  General Manager for World Orphan Drug Congress USA, Terrapinn

This week, the P4A team are podcasting from the World Orphan Drug Congress in Washington D.C. We speak to André Choulika,Chairman and CEO of Cellectis on the challenges of commercializing a CAR-T cell product and ethical concerns surrounding it, and Anna Bucsicsfrom the Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA) on their work supporting new biotech entrepreneurs.

Presenter: Joanna Fernandes

Contributors: André Choulika,Chairman and CEO of Cellectis; Anna Bucsics,Project Advisor to MoCA and Sophie Schmitz, Managing Partner, P4A

Producer: Aparna Krishnan

The team discuss the creation of a new health economics advisory committee in Spain expected to influence pricing and reimbursement decisions for drugs; and Japan’s approval of its first gene therapy and CAR-T therapy  - AnGes' HGF Plasmid and Novartis' Kymriah respectively. 

Presenter: Joanna Fernandes

Contributor: Max Rex

Producer: Aparna Krishnan

This week, we look at Ireland's access to orphan drugs as compared to rest of western Europe and the first outcomes deal for CAR-T cell therapies in Germany. 

Presenter: Joanna Fernandes

Contributors: Nader Murad, Ciaran Cassidy

Producer: Aparna Krishnan

This week, the P4A team discuss the spate of mergers and acquisitions involving Big Pharma companies in the gene therapy space. Particularly, we looked at the deals behind Roche's acquisition of Spark Therapeutics and Biogen's buyout of Nightstar. Also, Vertex's continued struggle to get its cystic fibrosis drug Orkambi reimbursed in the UK. 

Presenter: Joanna Fernandes

Contributor and Producer: Aparna Krishnan

On Rare Disease Day, P4A discusses the role of policy-makers in the healthcare system and specifically, the issues surrounding cross-border healthcare as part of its '6P' campaign.   

This directive is a key cornerstone legislation by EU officials that enables patients to find treatment in healthcare facilities outside their home country. However, there are several challenges associated with it. 

Presenter: Christina Poschen

Contributor: Dr Andrzej Rys, Director - Health Systems and Products

Producer: Aparna Krishnan

In another special edition episode dedicated to the '6P' campaign to mark Rare Disease Day, the Weekly RoundUp team discusses the pharmaceutical perspective on partnerships in the rare disease space.  

Presenter: Max Rex

Contributor: Jan-Willem Schmitz, General Manager for Nordics and Baltics, Sanofi Genzyme

Producer: Aparna Krishnan

 Throughout February, Partners4Access is running a campaign to mark the annual Rare Disease Day on February 28. The '6P' campaign is aimed at creating awareness on the impact of rare diseases on key stakeholders and the need for partnership to achieve successful treatment access for patients. The 6Ps are Partnership, Patient, Policy-maker, Physician, Pharma and Payer. This week we focus on the payer who are decision-makers assessing the value of a product in the healthcare system.  

Presenter and Producer: Aparna Krishnan

Contributor: Einar Andreassen, senior advisor at the Norwegian Medicines Agency. Einar is a health technology assessment analyst and reimbursement decision maker for the national insurance scheme in Norway.  

Throughout February 2019, the P4A team are hosting a series of special edition podcast episodes to mark Rare Disease Day. The initiative is part of a new 6P campaign aimed at promoting awareness about rare disease challenges and its impact on people and society. The 6Ps are Partnership, Policy-maker, Payer, Pharma, Physician and Patient. Our message: 6Ps are essential to successfully serve the rare disease community and achieve access to medicines.

This week, we discuss the first P – Partnership. The team reflects on the meaning of partnership in the rare disease context and what it can achieve through examples of different types of collaborations. From patient and clinical experts influencing HTA decisions; key stakeholders coming together for the Hercules Project to a collaboration between US based ICER, Canada's CADTH and UK's NICE.

Presenter: Aparna Krishnan

Contributors: Sophie Schmitz, Christina Poschen, Max Rex, Joanna Fernandes, Nader Murad and Jack Rawson

 This week we are looking at the USFDA’s proposed plans to address the expected rise in cell and gene therapy product applications and a significant development in a new controversial procedure called gene drive.

Also,  P4A is starting a new campaign that will run throughout February to mark Rare Disease Day.  The '6P'  campaign is aimed at creating awareness on the impact of rare diseases on key stakeholders and the need for partnership to achieve successful treatment access for patients. The 6Ps are Partnership, Patient, Policy-maker, Physician, Pharma and Payer. P4A will roll out a series of podcast episodes featuring thought leaders representing these stakeholders. So make sure you listen in!

Presenter: Joanna Fernandes

Contributors: Nader Murad and Sophie Schmitz

Producer: Aparna Krishnan

This week, the P4A team discusses the Louisiana Medicaid program implementing the 'Netflix' subscription model to pay for hepatitis C drugs and the potential Brexit options facing the UK government. 

Presenter: Joanna Fernandes

Contributor: Max Rex

Producer: Aparna Krishnan

Happy New Year to Weekly Roundup listeners! 2019 is shaping up to be an exciting year for the healthcare and biotechnology industry. We are only a couple of weeks in and already news developments are buzzing on the regulatory and corporate front.

So for this week's episode, we start by discussing the recent wave of  mergers and acquisition deals announced by Big Pharma namely, Eli Lilly and Loxo; BMS and Celgene as well as GSK and Tesaro. Also, the P4A team look at the implications of the current US government shutdown on the FDA and the agency's initiative on assessments for innovative drugs. 

Presenter : Max Rex

Contributor : Joanna Fernandes

Producer: Aparna Krishnan